Synageva Grabs $25M for Rare Diseases Drug Pipeline

3/21/11

Synageva BioPharma has nabbed $25 million in private equity financing as the Lexington, MA-based firm makes progress with its pipeline of treatments for rare diseases—an area that seems to be white hot these days, given Sanofi-Aventis’ (NYSE:SNY) $20.1 billion buyout of Genzyme (NASDAQ:GENZ) and other recent developments.

Synageva says that its previous lead investors participated in the new round of financing, which brings the total it has raised in the past two and a half years to $70 million. In 2009, the firm raised $45 million in a Series F round of financing from New Leaf Venture Partners, Hunt Ventures, Yasuda Enterprise Development, Baker Brothers Investments, Tullis Health Investments, and Four Rivers Partners, according to Venture Deal.

The company, which has several rare disease treatments in its pipeline, has an enzyme-replacement therapy code named SBC-102 in early clinical development for lysosomal acid lipase deficiency (a category that includes Wolman disease and cholesteryl ester storage disease). As you might guess, the lead drug is intended to replace the enzyme called lysosomal acid lipase; deficiencies of the enzyme cause fatty materials called lipids to accumulate in tissues throughout the body, leading to gastrointestinal, liver, and cardiovascular complications, according to the company.

“This latest funding further supports our mission of improving the lives of patients suffering from devastating rare diseases,” said Sanj Patel, Synageva’s president and CEO, in a statement.

While lysosomal storage disorders such as lysosomal acid lipase deficiency are relatively uncommon, treatments for them have been the bread and butter of companies that specialize drugs for rare diseases. For example, Cambridge, MA-based Genzyme’s top sellers include treatments for the lysosomal storage disorders Gaucher and Fabry diseases. Shire Human Genetic Therapies, a Lexington, MA-based unit of Ireland’s Shire, also has drugs for those two diseases and one for at least one other disorder of this type, Hunter syndrome.

These treatments are extremely expensive, as there are often few if no other treatment options for patients with rare diseases. Genzyme, for one, gets around $200,000 per patient annually for its Gaucher drug imiglucerase (Cerezyme). Hungry for new products to sell, big pharmaceutical companies like Paris-based Sanofi and Pfizer (NYSE:PFE) have been making major pushes to gain stakes in the rare diseases game. Sanofi is expected to close on its purchase of Genzyme early in the second quarter of 2011.

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